The U.S. Food and Drug Administration approved Nexviazyme (avalglucosidase alfa-ngpt) for the treatment of patients 1 year of age and older with late-onset Pompe disease, a progressive and debilitating muscle disorder that impairs a person’s ability to move and breathe.
Data with Sanofi’s investigational enzyme replacement therapy avalglucosidase alfa in late-onset Pompe disease and infantile-onset Pompe disease will be featured as platform and poster presentations at the 17th annual WORLDSymposiumTM, to be held February 8-12, 2021.
Fast Track, Breakthrough and Priority Reviews: Sanofi and ViiV
Acid alpha-glucosidase (GAA) enzyme, Biologics License Application (BLA), Breakthrough Therapy Designation, Bruton's Tyrosine Kinase (BTK) Inhibitors, Chronic Immune Thrombocytopenia (ITP), Clinical Trials, Enzyme Replacement Therapy (ERT), Fast Track Designation, FDA, HIV pre-exposure prophylaxis (PrEP), HIV Prevention, Muscle Disease, Orphan Drug Designation, PDUFA, Pompe disease, Priority Review Status, R&D, SanofiThe U.S. Food and Drug Administration granted various designations for Sanofi’s avalglucosidase alfa for Pompe disease, Sanofi’s rilzabrutinib for immune thrombocytopenia, and ViiV Healthcare’s cabotegravir for HIV prevention.
San Diego-based Retrophin is buying Orphan Technologies for $90 million in cash up front and up to $427 million in milestone payments.